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# Draft Guide on Developing Early Stage Alzheimer’s Drugs
- URL: https://www.fdaweb.com/draft-guide-on-developing-early-stage-alzheimers-drugs/
- Published: 2024-03-11T12:00:00.000Z
- Updated: 2026-09-14T14:24:58.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5156521

FDA has released a [draft guidance](https://www.fda.gov/media/110903/download?ref=fdaweb.com) entitled “Early Alzheimer’s Disease: Developing Drugs for Treatment.” The document addresses drug clinical development for treating stages of sporadic Alzheimer’s disease (AD) that occur before the onset of overt dementia (Stages 1 through 3, which are also referred to as early AD).

The document says that as AD’s scientific understanding has evolved, “efforts have been made to incorporate in clinical trials the use of biomarkers reflecting underlying AD pathophysiological changes and the enrollment of subjects with AD at earlier stages of the disease, in which there may be minimal or no detectable abnormality on clinical assessments. These efforts are particularly important because there may be an opportunity to intervene very early in the disease process of AD, given the slowly progressive course of AD and the development of characteristic pathophysiological changes that greatly precede the development of clinically evident findings.”

FDA says that delaying or halting/reversing the pathophysiological process is the “ultimate goal of presymptomatic or very early symptomatic intervention,” and treatments directed at this must begin before there are overt clinical symptoms. “This opportunity carries with it the need to understand ways to assess treatment benefit in these earlier stages of disease.”

The guidance also says that clinical trial enrollment for treating early AD should be based on “current consensus diagnostic criteria intended to establish the true biological presence of AD rather than criteria based on syndromic or other definitions; this approach is intended to avoid enrollment of a substantial number of subjects who would not actually have AD.”

Additionally, FDA says it supports “biologically based diagnostic criteria that are grounded in a contemporary understanding of the pathophysiology and evolution of AD. The characteristic pathophysiological changes of AD precede, often by many years or even decades, the development of clinically evident findings and progress as a continuous disease process that can be categorized into stages.”

It further says that based on previous clinical trials and the evolving understanding of AD’s pathophysiology, there is an increased focus on evaluating drug treatments for AD in the earliest disease stages. “Diagnostic criteria that reliably define a population with early AD, including the earliest stages characterized only by pathophysiological changes, are suited to the evaluation of drugs intended to delay or prevent the emergence of overt symptoms,” it says.